Patients With MDS, Acute Myeloid Leukemia (AML) Evolving From MDS, and Chronic Myelomonocytic Leukemia (CMML)
Conditions
Brief summary
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival. The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
Detailed description
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival. The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
Interventions
DRUGCyclophosphamide
-Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2 days)
Sponsors
Asan Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
15 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* MDS defined by WHO classification, CMML, or AML evolving from MDS A. International Prognostic Scoring System (IPSS) \> 1.0 or bone marrow blast ≥ 5% at any time points before HCT or B. AML progressed from MDS or C. CMML with bone marrow blast ≥ 5% at any time points before HCT * Patients receiving first HCT * Patients with appropriate hematopoietic cell donors A. HLA-matched sibling donor B. Unrelated donor C. HLA-mismatched familial donor * 15 years old or older , under 75 years * Adequate performance status (Karnofsky score of 70 or more) * Adequate hepatic function (AST or ALT \< 3 x upper normal limits and bilirubin \< 1.5 x upper normal limit). * Adequate renal function (creatinine \< 2.0 mg/dL or creatinine clearance ≥ 50 mL/min) * Adequate cardiac function (left ventricular ejection fraction ≥ 50% on heart scan or echocardiogram) * Adequate pulmonary function: DLCO, FEV1, and FVC ≥ 45% predicted by pulmonary function tests * Signed and dated informed consent must be obtained from both recipient and donor.
Exclusion criteria
* Presence of significant active infection * Presence of uncontrolled bleeding * Any coexisting major illness or organ failure * Patients with psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible * Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception * Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years following therapy with curative intent (except curatively treated nonmelanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia) * Presence of contraindications to cyclophosphamide (patients receiving pentostatin, symptomatic cystitis, urinary tract obstruction, with a history of hypersensitivity reactions to the component of the drug)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| GVHD-free, relapse-free survival | 8 years | This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival. |
Countries
South Korea
Outcome results
None listed